Preclinical Advances of Therapies for Laminopathies
Laminopathies are a group of rare disorders due to mutation in <i>LMNA</i> gene. Depending on the mutation, they may affect striated muscles, adipose tissues, nerves or are multisystemic with various accelerated ageing syndromes. Although the diverse pathomechanisms responsible for lamin...
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MDPI AG
2021
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oai:doaj.org-article:1dac7776c7c14d8c90553a1911ea96312021-11-11T17:30:03ZPreclinical Advances of Therapies for Laminopathies10.3390/jcm102148342077-0383https://doaj.org/article/1dac7776c7c14d8c90553a1911ea96312021-10-01T00:00:00Zhttps://www.mdpi.com/2077-0383/10/21/4834https://doaj.org/toc/2077-0383Laminopathies are a group of rare disorders due to mutation in <i>LMNA</i> gene. Depending on the mutation, they may affect striated muscles, adipose tissues, nerves or are multisystemic with various accelerated ageing syndromes. Although the diverse pathomechanisms responsible for laminopathies are not fully understood, several therapeutic approaches have been evaluated in patient cells or animal models, ranging from gene therapies to cell and drug therapies. This review is focused on these therapies with a strong focus on striated muscle laminopathies and premature ageing syndromes.Louise BenarrochEnzo CohenAntonio AtalaiaRabah Ben YaouGisèle BonneAnne T BertrandMDPI AGarticle<i>LMNA</i>lamin A/CprogerinlaminopathytreatmenttherapyMedicineRENJournal of Clinical Medicine, Vol 10, Iss 4834, p 4834 (2021) |
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DOAJ |
language |
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<i>LMNA</i> lamin A/C progerin laminopathy treatment therapy Medicine R |
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<i>LMNA</i> lamin A/C progerin laminopathy treatment therapy Medicine R Louise Benarroch Enzo Cohen Antonio Atalaia Rabah Ben Yaou Gisèle Bonne Anne T Bertrand Preclinical Advances of Therapies for Laminopathies |
description |
Laminopathies are a group of rare disorders due to mutation in <i>LMNA</i> gene. Depending on the mutation, they may affect striated muscles, adipose tissues, nerves or are multisystemic with various accelerated ageing syndromes. Although the diverse pathomechanisms responsible for laminopathies are not fully understood, several therapeutic approaches have been evaluated in patient cells or animal models, ranging from gene therapies to cell and drug therapies. This review is focused on these therapies with a strong focus on striated muscle laminopathies and premature ageing syndromes. |
format |
article |
author |
Louise Benarroch Enzo Cohen Antonio Atalaia Rabah Ben Yaou Gisèle Bonne Anne T Bertrand |
author_facet |
Louise Benarroch Enzo Cohen Antonio Atalaia Rabah Ben Yaou Gisèle Bonne Anne T Bertrand |
author_sort |
Louise Benarroch |
title |
Preclinical Advances of Therapies for Laminopathies |
title_short |
Preclinical Advances of Therapies for Laminopathies |
title_full |
Preclinical Advances of Therapies for Laminopathies |
title_fullStr |
Preclinical Advances of Therapies for Laminopathies |
title_full_unstemmed |
Preclinical Advances of Therapies for Laminopathies |
title_sort |
preclinical advances of therapies for laminopathies |
publisher |
MDPI AG |
publishDate |
2021 |
url |
https://doaj.org/article/1dac7776c7c14d8c90553a1911ea9631 |
work_keys_str_mv |
AT louisebenarroch preclinicaladvancesoftherapiesforlaminopathies AT enzocohen preclinicaladvancesoftherapiesforlaminopathies AT antonioatalaia preclinicaladvancesoftherapiesforlaminopathies AT rabahbenyaou preclinicaladvancesoftherapiesforlaminopathies AT giselebonne preclinicaladvancesoftherapiesforlaminopathies AT annetbertrand preclinicaladvancesoftherapiesforlaminopathies |
_version_ |
1718432048120594432 |